Biomedicine Daily · Tuesday, 15 September 2026

Trastuzumab Deruxtecan Extends Median PFS to 14.3 Months as First-Line Therapy in HER2-Mutant Advanced NSCLC

In the phase III DESTINY-Lung04 trial, first-line trastuzumab deruxtecan (Enhertu) gave a median progression-free survival of 14.3 months in patients with HER2-mutant advanced non-small cell lung cancer, versus 8.3 months with pembrolizumab plus chemotherapy. It is the first HER2-targeted therapy to show superiority over standard treatment in this setting.

Ivonescimab Beats Pembrolizumab on Overall Survival in First-Line PD-L1-Positive Advanced NSCLC

Ivonescimab significantly extended overall survival versus pembrolizumab as first-line treatment for PD-L1-positive advanced NSCLC, meeting the prespecified statistical bar for significance. At a median follow-up of 36 months as of August 20, 2026, 234 overall survival events had occurred and median overall survival reached 30.8 months. Prespecified subgroup analyses showed a consistent survival benefit, most pronounced in patients with high PD-L1 expression.

Parallel Phase III Trials Show B7-H3 ADCs Cut Death Risk in Relapsed Small Cell Lung Cancer

Two Phase III trials of B7-H3 antibody-drug conjugates in second-line relapsed small cell lung cancer, TAISHAN-302 and ARTEMIS-008, both hit their primary overall survival endpoint against topotecan, and TAISHAN-302 was published simultaneously in the New England Journal of Medicine. Reported back to back at WCLC 2026, the trials yielded identical OS hazard ratios of 0.46 (95% CI 0.35-0.62), providing independent, replicable Phase III evidence that B7-H3 ADCs beat current standard treatment in this setting.

AstraZeneca Oral SERD Camizestrant Misses PFS Primary Endpoint in Phase 3 SERENA-4 First-Line Breast Cancer Trial

Camizestrant plus the CDK4/6 inhibitor palbociclib missed the primary endpoint of progression-free survival compared with anastrozole plus palbociclib in the phase 3 SERENA-4 trial, AstraZeneca announced on September 11. The randomized, double-blind study enrolled 1371 patients with ER-positive, HER2-negative advanced breast cancer receiving no prior systemic therapy for advanced disease, including 194 in mainland China, across 263 sites. The result weakens the case for the oral SERD as a first-line endocrine partner to palbociclib.

Gastric Residual Volume Assessment in Critically Ill Children: The GASTRIC-PICU Randomized Clinical Trial.

Not routinely assessing gastric residual volume in ventilated critically ill children was noninferior to 6-hourly assessments for survival and ventilator-free days at 30 days (median 25 days in both groups; adjusted OR 0.95 [95% CI 0.86-1.05]). In the trial of 4700 children in pediatric intensive care units in the UK and Switzerland, dropping routine GRV checks also improved feeding: mean energy requirements achieved by 72 hours were 80.3% versus 76.8% (adjusted mean difference 3.2 percentage points, P < .001).

Roche, MediLink notch phase 3 lung cancer win but trail GSK on cross-trial survival

A phase 3 trial of the Roche-partnered, B7-H3-directed antibody-drug conjugate from MediLink hit its primary endpoint in small-cell lung cancer. The median overall survival benefit, however, fell short of the cross-trial bar set by GSK's rival B7-H3 asset, positioning MediLink and Roche behind GSK in this setting.

Scholar Rock scores long-awaited FDA approval, the first for muscle loss in SMA

FDA Accepts Takeda's Zasocitinib NDA for Plaque Psoriasis With Priority Review

Takeda said the FDA has accepted for priority review its new drug application for zasocitinib (TAK-279), an oral TYK2 inhibitor in development for adults with moderate to severe plaque psoriasis. Takeda acquired the drug from Nimbus Therapeutics in 2022 for $4 billion upfront and up to $6 billion in total. A regulatory decision from the FDA is the next step.

STAT+: Trump’s secretive pharma deals may undermine ‘most-favored nation’ pricing, an analysis suggests

A new analysis suggests that secretive deals the Trump administration struck with more than two dozen pharmaceutical companies could cut projected savings from its 'most-favored nation' Medicare pricing plan by as much as 80%. The plan, which the White House says would save Medicare $26 billion over several years, would tie Medicare prices to those paid in 19 other wealthy nations through two pilot programs, GLOBE for Medicare Part B and GUARD for Part D, with additional rebates required when prices exceed the lowest international price. More detailed rules are due shortly.

Cellectis cans allogeneic CAR-Ts to hop aboard in vivo bandwagon

Creating bottom-up RNA transfer vehicles from synthetic protein assemblies.

AI-designed protein assemblies combined with natural protein domains yielded more than 100 bottom-up RNA transfer vehicles with unique sizes and shapes, surpassing the RNA transfer efficiency of widely used delivery vehicles by several orders of magnitude. Tropism was programmed with computationally designed peptide binders, enabling delivery of therapeutically relevant cargo RNAs across cellular models. One vehicle's in vivo biodistribution was mapped in a mouse at near-single-cell resolution, confirmed as safe, and used for a gene-editing treatment strategy for Duchenne muscular dystrophy in patient-derived cells and a pig.

Copper ion-induced MYH9 polymerization executes cuproptosis.

MYH9 (myosin heavy-chain 9) acts as an executioner of cuproptosis: copper binding induces MYH9 polymerization, which disrupts the actin cytoskeleton and causes cuproptotic cell death. The compound HThPA triggers this process by binding the nuclear receptor Nur77, depleting the copper chelate glutathione and releasing free copper from intracellular stores. Physiological copper levels were higher in drug-resistant tumor cells and clinical samples, increasing their susceptibility to HThPA-induced cuproptosis, suggesting a treatment strategy for drug-resistant tumors.

A serpin-myeloid axis in pancreatic cancer heterogeneity and immune evasion.

SERPINE1 (PAI1) and SERPINB2 (PAI2) act as dominant organizers of immune evasion in pancreatic ductal carcinoma, building fibrin-rich extracellular matrix niches that programme macrophages toward immunosuppressive states and exclude cytotoxic T cells. Perturb-map spatial genomics in mice traced this remodelling to early gene-driven changes that precede spatial clonal dominance. Loss of either serpin, or pharmacological inhibition of PAI1 or CD18, improved tumour control and synergized with anti-PD-1, while patient tumour analysis showed immunosuppressive niches around rare SERPIN-expressing subpopulations dominated by SPP1+/MARCO+ macrophages.

Copper ion-induced MYH9 polymerization executes cuproptosis

Copper binding drives polymerization of MYH9, causing cytoskeletal collapse and executing cuproptosis. HThPA depletes glutathione to release intracellular copper, exploiting this pathway as a therapeutic vulnerability in drug-resistant tumors. The work identifies MYH9 polymerization as the mechanistic link between copper accumulation and cuproptotic cell death.

Predicting genome-wide functional constraints with GPN-Star.

GPN-Star, a phylogeny-aware genomic language model that uses whole-genome alignments and species trees, outperforms previous methods across variant effect prediction tasks in coding and non-coding human genome regions. Trained on vertebrate, mammal and primate alignments, it improves prioritization of pathogenic and fine-mapped GWAS variants, yields strong complex trait heritability enrichments, and improves rare variant association testing power. Versions trained for five model organisms, from mouse to Arabidopsis, show the framework generalizes.

Amplification of RNA for identification of Zika and HCV in whole blood.

A blood drying protocol with thermal treatments and primer-limited reverse transcription stabilizes RNA in whole blood, allowing direct amplification without extraction, purification, or cold-chain logistics. Detection reached 10 copies per microliter for Zika virus and 1 international unit per microliter for HCV, and larger blood volumes achieved single-copy sensitivity without sample loss. Zika and HCV were detected with a portable fluorometer, supporting field-deployable point-of-care diagnostics.

Germline Variants Absent from Global Reference Databases in Malawian DLBCL Patients: Implications for Variant Interpretation in African Genomic Medicine

Whole-exome sequencing of 55 Malawian patients with diffuse large B-cell lymphoma identified 332 germline variants (0.26% of 127,456 PASS-filtered variants) absent from gnomAD, ExAC, and 1000 Genomes, distributed across 289 genes. Database-absent variants were enriched for predicted loss-of-function consequences (16.9% vs 7.8%; OR 2.41) and a substantial share occurred in HLA class I loci. The results show that global reference databases miss variants present in this population, creating uncertainty in clinical variant interpretation for African patients.

Dosimetric Impact of Residual Intrafraction Motion in Gated MR-Guided Prostate SBRT With Focal Dose Intensification

Residual intrafraction motion during gated MR-guided prostate SBRT with simultaneous integrated boost caused directionally asymmetric coverage loss, hitting the boosted GTV hardest: per-fraction median GTV deviations were −1.2% for D95 and −5.6% for V45Gy across 150 fractions in 30 patients. Motion-inclusive dose reconstruction yielded asymmetric internal target volume margins (2/2/1/2/1/2 mm for GTV) that achieved sufficient coverage in about 94% of fractions per direction, at the cost of an average 6.9 ± 4.7% reduction in gating duty cycle.

An engineered nanopore identifies saccharides, amino acids, peptides and ribonucleotides

The paper describes an engineered nanopore that identifies saccharides, amino acids, peptides and ribonucleotides. Machine learning classifiers, including a general classifier and few-shot learning approaches, were trained to identify these molecule classes from nanopore signals. The data and custom code are provided with the paper.